Research Interests

The Disney group is focused on developing rational and predictable approaches to design highly selective therapeutics from only genome sequence. One of the major articulations of the utility of genome sequencing efforts has been in advancing patient-specific therapies, yet such developments have been only sparsely reported.

We accomplish this lofty goal by using advancements in annotating RNA structure from sequence and several novel technologies that we have recently developed in our laboratory. Our current focus is on leveraging these technological advances to identify patient-specific therapies targeting orphan diseases, especially RNA-mediated neurological disorders, with no known cure or more common disorders to which there is a poor prognosis, such as drug resistant Cancers.

Developing specific lead therapeutics that reduce the production of toxic proteins that are known to cause the majority of cases of Amyotrophic Lateral Sclerosis (ALS, Lou Gehrig’s disease) and Frontotemporal Dementia (see: Neuron. (2014), 83, 1043-50. doi: 10.1016/j.neuron.2014.07.041)

Developed chemical approaches to allow for a diseased cell to synthesize its own drug, allowing new precision medicine strategies. (see: Angewandte Chemie International Edition English(2014), in press. doi: 10.1002/anie.201406465)

Exploiting important classes of drug targets in multiple disorders that are viewed as being impossible to “drug.”

Developing and implementing novel technologies that allow for the precise reaction and cleavage of RNA targets by using small molecules to both identify and further manipulate therapeutically relevant RNAs by small molecules.